My hope for the lockup is that there are enough anxious sellers to create some cheap shares to be bought by long term buyers.
I'm comfortable with my holding but would love to pick up more shares at a cheaper price
First Cupid trial, many years ago:
"Only patients with undetectable neutralizing antibodies against AAV1 (titer
Was Mydicar the first "Breakthrough" that looked at a totally new type of therapy and say it should be given every opportunity to speedily move through the review process for the billions it could save our nations medical bill yearly? In addition to the fact that it has the potential to greatly improve the quality of the patients life.
FDA takes a close look at early data and grants "Breakthrough therapy" and agrees to an SPA that gives approval on basis of how soon a patient needs to be hospitalized post treatment.
expect dilution at $30 plus
Nothing should go wrong. Treated patients don't need to revisit the hospital after Mydicar treatment, unless the patient was too far gone at initiation.
Would love to see Celladon do another stock offer 5 millions shares at $30 partner off his product and take on the pulmonary indication. Would like a billion upfront. By the end of summer everyone running a trial site will have a very good idea why they have an arm of strong responders where there cardiac failure isn't spiraling to ever worse levels.
More leaks every week.
This one is going to save billions in health care dollars yearly.
Agree on possible buyout.
June 2, 2014
New CFO Paul Cleveland:
was Executive Vice President of Corporate Strategy and Chief Financial Officer of Aragon Pharmaceuticals, Inc. Aragon was sold to Johnson & Johnson in August 2013 for $1 billion.
Before the CUPID study....The analysis of multiple end points in early heart failure trials (versus a single primary end point such as ejection fraction [EF]) has also been suggested recently by the Food and Drug Administration in a cross-center guidance document pertaining to cellular therapy for cardiac disease.
After the FDA had a serious look at the date in the first Cupid Trial:
Celladon has obtained a Special Protocol Assessment (SPA), whereby the U.S. Food and Drug Administration (FDA) has agreed to use time-to-multiple heart failure-related hospitalizations in the presence of terminal events
And, granted "Breakthrough Therapy" for this SPA endpoint.
dawg, much appreciated. Been doing a lot of due diligence trying to poke some holes in what looks like a grossly undervalued company. Staying impressed, so far.
Looking at ages of each arm in the trial
Placebo - 61.0
Low Dose - 60.3
Mid Dose - 63.9
High Dose- 56.6
Would like to know how the high dose average skewed so much younger? Do some adjustments need to be made when comparing outcome of 56 yo patients to a 61 yo group? I'm sure there were some 60 year olds in the high dose arm and would love to know how they are faring.
This one will also save insurance companies on the rehospitalization cost, billions saved yearly. Should be easy to get Medicaid and Isurance to start paying. The Insurance Industry would also like to see even earlier intervention research to start ASAP?
Affordable and as an outpatient treatment. Could lead to heavy worldwide demand where an experienced partner like Roche may be willing to pay a HUGE upfront fee and royalty stream for distribution outside of US?
A huge improvement in patient outcome and affordable? Worldwide sales could become a serious focus and that would lead to partnering.
SCRIP Intelligence article
Celladon president and CEO Krisztina Zsebo:
KZ: Cost of goods is not going to be an issue. It's an important consideration and we have three strategic pharma investors – Pfizer, Novartis and Johnson & Johnson – and I can assure you that we endured the full extent of their diligence efforts on all aspects of our technology for many, many months at a time. I feel that we're on very solid footing with regards to the production and cost of goods.
investor relations Fredrik Wiklund:
We are working with Lonza, who are one of the absolute leaders in the industry of producing mass-scale biologics. We estimate that when we will be in full commercial mode and producing this, the cost of goods or the drug cost itself is really quite modest, probably $1,000 to $2,000 per dose per patient. When you put that in the context of what typical biological drugs cost on an annual basis, it's moderate.
Agree with your edit of my post in defining end stage. I do think the FDA will need to think hard about making Mydicar an option for the early stage Class 4 and Class 3 patients. Just need some strong responders in Cupid 2 to make the case
The enlarged heart population could be aiming for much earlier intervention and much larger patient population.
“In general the term ‘enlarged heart’ refers to heart failure,” said Clyde Yancy, M.D., past president of the American Heart Association and chief of the Division of Cardiology and the Magerstadt Professor of Medicine at Northwestern University Feinberg School of Medicine in Chicago. “This is a common condition that’s more likely to occur in older patients. It’s most strongly related to a history of high blood pressure or a previous heart attack.”
About one in five adults over age 40 is affected, with African-Americans facing a greater risk.
“There are other reasons for an enlarged heart or heart muscle disorders (like cardiomyopathies, which are diseases of the heart muscle) and not everyone with heart failure has an enlarged heart,” Dr. Yancy said. “But when we speak of an enlarged heart we are typically referring to heart failure.”
* Does Celladon sell their first drug for a few billion if the news on Cupid is good? Hope not.
Early availability approval for end stage patients?
In 2012, we obtained a Special Protocol Assessment, or SPA, whereby the FDA agreed to use time-to-multiple heart failure-related hospitalizations as the primary endpoint for a MYDICAR Phase 3 pivotal trial. Our ongoing CUPID 2 trial uses a similar clinical protocol with identical endpoints as agreed to in the SPA.
...The one- and six-month readmission rates after heart failure-related hospitalization are close to 25% and 50%, respectively, and there is growing pressure on hospitals to reduce readmissions for heart failure.
(2014 Mydicar gets Breakthrough Therapy status) - No idea why this company doesn't have a market cap much higher as Cupid 2 leaks should be obvious to the big boys following this trial.
GLTA, missed todays dip
Assistance Publique – Hopitaux de Paris primary endpoint ((six months))
My wild guess is that some previous trial patients were super responders in the first Cupid trial.
Assistance Publique – Hopitaux de Paris
dawg, another partner was impressed, in something they saw, and is conducting a trial to see if Mydicar can actually reverse some of the damage done to the heart by HF:
Celladon also announced that investigators have initiated the AGENT-HF Trial (AAV1-CMV-SERCA2a Gene Therapy Trial in Heart Failure). This trial is an investigator-initiated clinical trial and is partially funded by the French government and sponsored by Assistance Publique – Hopitaux de Paris. This trial is not required by any regulatory authorities for systolic heart failure indications, but the patients enrolled in this trial will be included in the overall safety database submitted in an MAA submission.
The primary objective of the AGENT-HF Trial is to determine whether treatment with MYDICAR leads to reverse remodeling of the heart. In patients with heart failure, the size, shape, structure and physiology of their heart changes over time, and these changes that lead to a progressive decline in left ventricular (LV) function are referred to as remodeling. In reverse remodeling, there would be changes back to the more normal, healthier state of the heart along with an improvement in the functioning of the heart. This trial will enroll approximately 44 heart failure patients in France with half receiving MYDICAR and the other half placebo. The primary endpoint at six months will be change, compared to baseline, in LV end systolic volume as measured by cardiac computed tomography.
(I suspect they will see improved function regardless of actual physiological turning back the clock. Maybe the organization has looked at data in a completed Celladon study and was impressed at some of the patients data they saw?)
Found an interesting take on the first Cupid trial
" Molecular Therapy" 2011
CUPID is a phase II randomized, double-blind, placebo-controlled trial that evaluated the effectiveness of a gene transfer vector based on adeno-associated virus 1 (AAV1) for delivery of SERCA2a complementary DNA in 39 patients with advanced heart failure.
There are several reassuring points in Jessup and colleagues’ data set. One is that this is the first clinical application of AAV vectors for cardiac gene transfer. By virtue of its exquisite and still largely unexplained capacity to transduce postmitotic cells, AAV is currently the vector of choice for cardiac applications. Second, it is interesting to learn that a single intracoronary infusion of the vector has been sufficient to provide a potential therapeutic benefit in a condition where the transgene must be expressed inside the cells and thus where a high efficiency of transduction is required. In this respect, the authors have taken advantage of AAV serotype 1
(Lots more detail in the article. It looks like a big endorsement that the first Cupid trial has shown cardiac gene therapy is more than scientific fantasy)
The Big Boyz have their ways of following trials and finding out about trends. Could be happening here?
The current 250 person trial has been going on for a few years and the control arm will likely be revealing itself early. The Journal of American College of Cardiology State-Of-The-Art Paper | January 2013:
" Patients with hospitalization for HF (HHF)....Discharge from a heart failure hospitalization is followed by a readmission within 30 days in ≈24% of cases."
Mydicar patients may be showing significant improvement in the 30 day post discharge reporting? If so, it's likely the "breakthrough" status is drawing interest in what the Data Monitoring Committee (DMC) is seeing.
* Just some wild guessing on why so few shares are up for sale at what has been very nice percentage gains. GLTA, this one has the potential for huge returns if all goes well.